By Cam Lucadou-Wells
A Berwick woman’s campaign to put a life-saving medication in affordable reach for stricken families has been successful.
Julie Cini, the head of the Hallam-based group Spinal Muscular Atrophy Australia, represents more than 200 families whose children suffer the cruel, rare disease SMA.
She was “extremely excited” that the “phenomenal” drug Spinraza or Nusinursen was recently recommended for listing on the Federal Government’s Pharmaceutical Benefits Scheme.
It is otherwise prohibitively expensive – about $750,000 for a 12-month course of six injections.
“We eagerly await Minister (Greg) Hunt’s announcement of this (PBS) listing date,” Ms Cini said.
In the interim, pharmaceutic maker Biogen will offer the medication free to patients that meet the PBS criteria.
The PBS scheme will benefit patients under 18 and who have had symptoms of SMA since younger than three. That leaves a large “unmet need” for adult patients, Ms Cini says.
“SMA Australia realise this part of our community will be devastated.
“Due to the infancy of the drug and lack of robust clinical data, the Pharmaceutical Benefits Advisory Committee (PBAC) was unable to assess the extent of the meaningful benefit the drug may have on the adult population.
“We encourage the adult population to contact us so we are able to better present your case to the government.”
She said the drug has “phenomenal” properties on children, halting the disease’s degenerative march and increasing motor-function.
Without the drug, the illness destroys motor neurons and muscles – the means for children to crawl, walk, feed, sit, cough, swallow and breathe.
“Normally type-1 SMA children have only 12 months of life,” Ms Cini says.
“Children on this treatment are now seeing their second birthday and they’re growing stronger.”
Holt MP Anthony Byrne tabled a 11,201-signature petition in Parliament to fast-track the drug after it was rejected for PBS listing in December.
“The recommendation would see paediatric patients with Types 1, 2 and 3a SMA be able to access this drug at a vital age before the severe degenerative effects of the disease take hold.
“Julie’s determination to promote this drug through the media, petitioning and meetings with Parliamentarians is an example of our Parliament at its best.
“It’s been a long journey which would not have been possible without the effort of a community champion such as Julie.”
